701-201: Respiratory Muscle Strength in Patients with Late-Onset Pompe
Research type
Research Study
Full title
Protocol 701-201: A Study of Respiratory Muscle Strength, including Effort-Independent Measures, in Subjects with Late-Onset Pompe Disease.
IRAS ID
160147
Contact name
Michael Polkey
Contact email
Sponsor organisation
BioMarin Pharmaceutical, Inc
Clinicaltrials.gov Identifier
2014-002158-38, European Clinical Trials Database (EudraCT) Number:
Research summary
This is an observational study which aims to better understand different measures of respiratory muscle strength in patients with Pompe disease. In a separate larger study, the effect of an experimental treatment for Pompe disease on respiratory muscle strength measured as Maximum Inspiratory Pressure (MIP) is being evaluated by change in MIP. However because MIP is a voluntary test it is subject to variability due to factors such as fatigue, illness, motivation.
Thus the aim of this study is to better understand the relationship of MIP with other measures of inspiratory muscle strength, in particular the pressure generated by the diaphragm when the phrenic nerves are maximally stimulated (the pressure from a "twitch" of the diaphragm or TwPdi), since this measure is independent of patient effort or aptitude. However, TwPdi is difficult to perform and not practicable at multiple centers where the separate treatment study is being performed. This study is being performed to support the use of MIP as the primary endpoint in the treatment study.
MIP is measured through a rubber mouthpiece connected to a pressure sensor. TwPdi is measured from pressure within two thin tubes, each passed through the nose: one into the oesophagus above the diaphragm, one into the stomach below the diaphragm. The phrenic nerves are stimulated by a magnetic field, and the difference in pressures generated between above and below the diaphragm is measured.
Ancillary tests of respiratory muscle function and of lung volumes as detailed in the protocol will be measured to provide a complete picture of respiratory muscle function and its physiological sequalae. These tests are in routine clinical use, albeit in specialised centres, for the evaluation of diaphragm function in man
In this study there will be no study treatment, by medication, exercise or physical treatment.REC name
North of Scotland Research Ethics Committee 1
REC reference
14/NS/1032
Date of REC Opinion
3 Sep 2014
REC opinion
Further Information Favourable Opinion